In January 2005, the oversight responsibility of the Post-Approval Studies Program was transferred to the Division of Epidemiology (DEPI) of the Office of Surveillance and Biometrics (OSB)/Center for Devices and Radiological Health (CDRH).
The CDRH Post-Approval Studies Program encompasses design, tracking, oversight, and review responsibilities for studies mandated as a condition of approval of a premarket approval (PMA) application, protocol development product (PDP) application, or humanitarian device exemption (HDE) application. The program helps ensure that well-designed post-approval studies (PAS) are conducted effectively and efficiently and in the least burdensome manner.
CDRH has established an automated, internal tracking system that efficiently identifies the reporting status of active PAS studies ordered since January 1, 2005 based on study timelines incorporated in study protocols and agreed upon by the CDRH and applicants. This system represents CDRH's effort to ensure that all PAS commitments are fulfilled in a timely manner.
In addition, CDRH launched this publicly available webpage to keep all stakeholders informed of the progress of each PAS. The webpage displays general information regarding each PAS, as well as the overall study status (based on protocol-driven timelines and the adequacy of the data) and the applicant's reporting status for each submission due.
This is a prospective, multicenter, single arm study with a total of 35 newly enrolled
patients, treated at 3 to 10 clinical centers in the United States. The primary objectives of this study are to evaluate the safety and probable benefit of the Liposorber LA- 15 System in relieving nephrotic syndrome associated with refractory pediatric primary FSGS at 1 month after the final apheresis treatment.
Study Population Description
Pediatric patients with nephrotic syndrome associated with primary focal segmental glomerulosclerosis, when the standard treatment options,
including corticosteroid and/or calcineurin inhibitors treatments, have been unsuccessful or not well tolerated, and the patient has a GFR ¡Ý 60 ml/min/1.73m2, or the patient is post renal transplantation.
A total of 35 patients will be included. The primary probable benefit endpoint will be assessed
using a 95% confidence interval. The assumption for sample size calculation was that 50% of patients would achieve a 50% reduction in urine protein at 1 month. Optimal conservative medical therapy would be expected to be approximately 10% based on the collected data in Glomerular Disease Collaborative Network (University of North Carolina, Chapel Hill, NC). The reported clinical results in pediatric steroid- resistant FSGS treated with the Liposorber® LA-15 System suggest the incidence of favorable cases is at least 25%, implying that the probable benefit of the system is superior to that of ordinary medical therapies. With a performance goal of 25%, an expected rate of 50% of patients, a one-sided exact binomial test, and a type I error rate of 0.025 (corresponding to a 95% one-sided confidence interval), 30 patients provides a power of 0.82.
The primary probable benefit endpoint is the percent of patients who show complete or partial remission
at 1 month after the final apheresis treatment. The primary safety endpoint is the rate of device-related and procedure-related serious adverse events (SAEs) occurring during the period in which the apheresis procedures are administered and up to at the 1-month follow-up visit. Secondary endpoints are as follows: - Nephrotic condition (complete remission, partial remission, and nephrotic state) at 1, 3, 6, 12, and 24 months after the final apheresis treatment, including the percentage of patients who obtain complete or partial remission at 3, 6, 12, and 24 months - Incidence of adverse events encountered during the period in which apheresis treatments are given - Incidence of all AEs and SAEs occurring within 3, 6, 12, and 24 months after the final apheresis treatment - Laboratory values, including eGFR, at baseline, after the last treatment, and at 1, 3, 6, 12, and 24 months after the final apheresis treatment, including percent change from baseline and percentage of patients showing an increase or decrease in each value
Followup Visits and Length of Followup
Patients will be followed for 24 months after they complete the apheresis procedures. Study visits will
be as follows: Pre-procedural exams and test, approximately 9 weeks of study apheresis procedures, and 1-, 3-, 6-, 12- and 24-month follow-up office visits.
New Enrollment PAS for the Liposorber LA-15 System